We are pleased to have completed the 3rd annual Women & MPN conference on Friday, September 29th, in Los Angeles. We had wonderful speakers and a lively panel discussion on issues impacting women and all those living with MPNs. One recurring theme was revealed by the newly diagnosed attendees; their physicians didn’t seem to know a lot about their ET, PV or MF and gave little support or direction to resources. MPN Advocacy & Education International is committed to a more vigorous outreach program to physicians and will continue to provide direction, education and resources to all patients and caregivers lacking the support they need. Our special guest, Emmy-award winning actress, Finola Hughes, will be a great spokesperson for the MPN community because of her ability to reach much greater numbers through the media.

While participating in the Women & MPN™ conference in Los Angeles at the end of September, I met several women who were newly-diagnosed with a myeloproliferative neoplasm. They are bright, can-do women who recently learned what is causing their crazy symptoms and cost some of them their employment. As I listened to their stories, a rush of memories ran through me:

Ten years ago, after severe abdominal thromboses, sepsis, and organ failure, a hematologist told me, “At least you don’t have cancer. You should see some of my really sick patients. When you recover from the surgery, you should start working out and lose some weight. You’ll feel better.” Eight years ago, when I almost stroked with a hematocrit of 69.1, another hematologist started treatment (phlebotomies and hydrea) and encouraged me to consult with a MPN expert. That expert noted some CBC results from several years earlier (separate health care organization) that showed platelet count over 900,000. He explained that I likely started with ET; it wasn’t diagnosed back then because they didn’t know as much and the internist didn’t connect my symptoms to a blood disorder.

Incredibly, in 2017, some hematologists still don’t understand the very diseases they diagnose. I get it; there’s a lot to know. Given the complexities of blood cancers and diseases, they have a responsibility to acknowledge their limitations and refer patients to specialists. Unfortunately, many newly-diagnosed MPN patients receive partial information, treatment plans that look more like Swiss cheese, and faulty expectations.

I naively assumed a decade after my first experience, MPNs would not be foreign to local practice physicians. In the last ten years, significant progress was made with new mutations identified, patient studies, new treatments, and diagnostic and treatment protocols. MPNs receive attention at American Society of Hematology (ASH) and other related conferences. I thought greater awareness and understanding would filter through the hematology network beyond university and research centers. Based on recent conversations with several new patients, this is not happening. And people in smaller communities are at greatest risk.

Fortunately, these women took the information from their physicians and started researching on their own. One way or another, they found MPN Advocacy & Education International’s conference and decided to attend. The physicians who spoke at the conference shared timely information in relatable ways. “I can’t tell you how much relief I feel! Meeting other patients and talking to doctors who know what they are talking about gives me hope,” remarked an attendee.

Another shared, “My doctor told me that PV is no big deal. Then I looked it up and read it is cancer and could kill me. After listening to all the speakers, I’m starting to believe that we can get a handle on this after all. I don’t understand half of what they said, but at least I know it’s complicated and treatable. Now I need to find a good MPN doctor!”

Don’t fight a fact. Deal with it!

So as the MPN progress train lurches forward, we must remember that everyone isn’t on board. Let this be a reminder to take charge of our care. Ask questions. Seek second opinions. Connect with others. Be mindful. Don’t give up. This caring MPN community is here for you.

How to Approach the Conversation with Your Child’s Hematologist

By Nicole Kucine, MD, MS
It can be difficult to know what to expect when your child is being evaluated for an MPN. There is limited guidance on the internet, which can make specialist visits overwhelming to families. As parents, you can expect that you and your child will be asked many questions about symptoms he or she might be having, including headache, abdominal symptoms, rashes, and itching. Your child will undergo a number of blood tests, including some genetic tests, as well as a bone marrow examination. Children who are having symptoms specific to a certain body area may need radiologic tests (such as an ultrasound or MRI.) You should feel free to ask anything you want, and make sure if you are searching the internet you look at reliable sources (such as the MPN Research Foundation, MPN Advocacy Education International, or cancer.gov.) Some of the questions I am often asked include the following:

Does my child have cancer?
This is a tricky question, and others may disagree, but I do not think of children with MPN as having cancer. In adults, the World Health Organization criteria considers MPN to be chronic forms of leukemia. We are still evaluating MPN in children, and at this point it is not clear that they are all the same disorders in kids as they are in adults. I view the classical MPN in children as chronic bone marrow disorders, and while they have the potential to transform to acute leukemia, this is not something that has been reported in the literature. By envisioning these as chronic illnesses, I think it helps to set the expectations for long-term follow-up and aiming for keeping day to day activities as normal for your child as possible.

Does my child need the bone marrow evaluation?
The answer to this is definitely yes. The bone marrow exam is an extremely important part of the diagnostic process. It can provide a lot of information about what is going on with your child’s blood cells at the source. Things like storage iron, fibrosis, and the appearance of the precursor blood cells are studied. The procedure itself is performed with anesthesia to make sure your child is asleep during the bone marrow test and doesn’t remember it. The pain following a bone marrow test is generally very mild, and children may not require any pain medicine or might require a dose of Tylenol.

What type of MPN does my child have?
The diagnostic criteria for the various types of MPN are based on years of study and data on adult patients. They include features like appearance of bone marrow cells, genetic findings, and lab criteria. Making the appropriate classification for adult patients is important for discussions of prognosis and treatment. We do not yet know if we can directly apply these criteria to children, and knowing the exact type of MPN each child has may not be possible. While it is important to gather all of this information in children, it may not be as important to specifically name the exact type of MPN, and “MPN, unclassifiable” is an appropriate diagnosis for a number of children. The decisions about how to counsel families, what treatments may be recommended, and what follow-up is needed, will be made based on a variety of findings.

Does my child require treatment for his or her MPN?
The answer to this question varies, as I do not believe treatment is required for all children with MPN. I usually determine the need for treatment based on an individual child’s symptoms and lab findings. I generally do not recommend treatment for children who are asymptomatic and have reassuring labs. Children with mild symptoms in the setting of a high platelet count can often benefit from low-dose aspirin, as long as they are not showing evidence of bleeding or acquired vonWillebrand disease. Children with high red blood cell counts can also benefit from low-dose aspirin or phlebotomy. When a child has a severe clinical event such as a blood clot, or does not have improvement of symptoms with initial therapy, then cytoreductive therapy is appropriate. Which medication is used should be decided based on a conversation with the family and the treating doctor. I have been asked about what is my “cutoff” for high platelet or red cell counts for treatment, and there isn’t a standard cutoff. For example, I don’t think an asymptomatic child with a platelet count of 1.3 million necessitates treatment if they are feeling well and otherwise healthy. If you asked 10 different hematologists, you would probably get 10 different opinions on when to treat, so I think it’s important to have a conversation with your hematologist about the risks and benefits of different treatments.

While it can be frustrating to be facing a rare disease, there is ongoing research to help us better understand these conditions in children and adolescents. Keeping an open mind and making sure you have good communication with your child’s hematologist is the most important thing you can do.

MPN Advocacy & Education International was invited to attend a very important and timely meeting with the FDA last month. The invitation was extended to us and a few other MPN focused organizations. This meeting launched a unique beginning for those of us representing the MPN Community.

The FDA’s newly formed Oncology Center of Excellence and the Office of Patient Outcomes are committed to engaging MPN patients and advocates to better respond to the need for quality treatment options.

Among the many topics presented by their hematologists, researchers and cancer patient liaison, was an in depth explanation on drug approval processes and how valuable and critical the patient’s voice is to their mission.

After a drug is approved, companies are required to send frequent updates on any and all reported adverse side effects from their drug. Physicians also report on these events and patients can do so by contacting the FDA directly through Med Watch Consumer Voluntary Reporting. Go to www.fda.gov/reportinghelp. Patient Reported Outcomes are extremely important and valuable to the Drug Approval Process and after a drug is approved.

We also learned the complexities surrounding drug “holds” and what happens and why when that hold begins and when it is released. They stressed the importance of ongoing communication between BioPharma and the FDA and encourage regular conversations and frequent updates with comprehensive data.

As the MPN Community pursues the use of Interferon and hopes for accessibility to Interferon as a viable option for treatment, we learned that company(ies) producing Interferon would have to apply for approval specifically for MPNs. It could be a daunting process but there may be avenues we can utilize through patient advocacy efforts.

Although we did not share all of our concerns at this meeting, we look forward to subsequent meetings where we hope to tackle the issues of endpoints, pricing, fast-tracking, and other challenges surrounding MPN drug approval. We were very pleased to have had this opportunity.

Individuals living with Essential Thrombocythemia, Polycythemia Vera and Myelofibrosis are now invited to participate in the first ever patient advocacy driven registry for people with an MPN (myeloproliferative neoplasm). This new initiative is called myMPN and was launched in September at the beginning of Blood Cancer Awareness Month. Information about this project and the link to enroll can be found at http://www.mpnresearchfoundation.org/myMPN.

Through myMPN the MPN Research Foundation provides patients with an opportunity to share information about their experience with MPN with researchers. The “How do you feel today” survey records symptom data in real time. There is a “Health Event” survey for people who have had a change in diagnosis, medication change, hospitalization or other medical event they’d like to include in myMPN. Participants will be able to see how they compare to others in myMPN. Almost 100 people are already participating on a regular basis.

Dr. Ruben Mesa is the chair of myMPN’s steering committee which also includes representatives from Mayo Clinic, Guys and St. Thomas’ Hospital in London, Mt. Sinai School of Medicine, Oregon Health Sciences University, MD Anderson and Johns Hopkins University. The Steering Committee provided feedback and oversight for the protocol and surveys that are included in myMPN and will help set data access guidelines and lead the evolution of the surveys over time.

NEW YORK, Aug. 30, 2017 (GLOBE NEWSWIRE) — Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel therapeutics for difficult to treat cancers, announced today that data from SL-801’s ongoing Phase 1 trial in patients with advanced solid tumors has been selected by the European Society of Medical Oncology (ESMO) for poster presentation at their Annual Congress 2017, to be held September 8-12, 2017 in Madrid, Spain. SL-801 is a novel, potent and reversible Exportin-1 (XPO1) inhibitor.

How do I know if I’m having ET symptoms or menopause symptoms, i.e. night sweats, foggy brain etc.?

There is no one answer for this – and many of the symptoms of essential thrombocythemia may be confused with “normal” symptoms of menopause. However, there are some tell-tale signs that ET is the root cause. For example, night sweats are different than hot flashes. Night sweats typically occur while sleeping and characteristically cause soaking sweats – like you have to change your pajamas or the bedding. Night sweats are not subtle. In contrast, most women describe hot flashes as a flush that comes on at any time of the day (or night) and while uncomfortable, doesn’t typically cause profuse sweating. If you are having migraine headaches, visual auras, painful rash of the extremities — called “erythromelalgia,” these are more common with ET and warrant a discussion with your physician.

Sex is difficult since my diagnosis. My husband is cautious and doesn’t want to impose himself on me, and I cannot get my head into it. I’m consumed with thoughts of the disease and possible progression. Can you offer an insights?

Many individuals, when faced with a life changing diagnosis, find that anxiety and depression about the diagnosis can affect the normal activities of life, whether or not it’s sexual activities or libido, work, sleep or other relationships. It is very, very common for people to experience grief reactions or event depression with diagnosis. Depression and anxiety are very treatable conditions.

In your situation, I would strongly recommend seeking out a psychotherapist or psychiatrist and discussing that your worries about your disease are impeding your normal function. You might also have your husband join you at some of these sessions. Finally, don’t forget to mention this to your hematologist as well, who might be able to give some advice with regard to a medical approach. The most stressful things in life: death, divorce, illness often cause humans to experience grief, which can also take the form of anxiety or depression. There is excellent treatment for this in the form of therapy and medication. Please talk to a professional.

I know intimacy is many things unrelated to sex but sometimes the fatigue overtakes me and I simply don’t care if I ever have sex. I also fall asleep when we try to be close.

Fatigue is a very common symptom of the MPNs, including the “lower-risk” MPNs including polycythemia vera and essential thrombocythemia. This can impede normal sexual activity and libido. One option is to try creative, non-medical, interventions. Think about what times of the day you are at your best. Perhaps timing your intimate encounters to be at a time of day when you feel more energized? Experiment with alterations in your diet – are there some foods that make you sleepy? If so, you can avoid those when you want to remain energized. Certainly, avoiding alcohol might help to prevent fatigue on “date nights.” A lack of libido can also be a sign of depression and certain

How do I know if I’m having ET symptoms or menopause symptoms, i.e. night sweats, foggy brain etc.?

There is no one answer for this – and many of the symptoms of essential thrombocythemia may be confused with “normal” symptoms of menopause. However, there are some tell-tale signs that ET is the root cause. For example, night sweats are different than hot flashes. Night sweats typically occur while sleeping and characteristically cause soaking sweats – like you have to change your pajamas or the bedding. Night sweats are not subtle. In contrast, most women describe hot flashes as a flush that comes on at any time of the day (or night) and while uncomfortable, doesn’t typically cause profuse sweating. If you are having migraine headaches, visual auras, painful rash of the extremities — called “erythromelalgia,” these are more common with ET and warrant a discussion with your physician.

Sex is difficult since my diagnosis. My husband is cautious and doesn’t want to impose himself on me, and I cannot get my head into it. I’m consumed with thoughts of the disease and possible progression. Can you offer an insights?

Many individuals, when faced with a life changing diagnosis, find that anxiety and depression about the diagnosis can affect the normal activities of life, whether or not it’s sexual activities or libido, work, sleep or other relationships. It is very, very common for people to experience grief reactions or event depression with diagnosis. Depression and anxiety are very treatable conditions.

In your situation, I would strongly recommend seeking out a psychotherapist or psychiatrist and discussing that your worries about your disease are impeding your normal function. You might also have your husband join you at some of these sessions. Finally, don’t forget to mention this to your hematologist as well, who might be able to give some advice with regard to a medical approach. The most stressful things in life: death, divorce, illness often cause humans to experience grief, which can also take the form of anxiety or depression. There is excellent treatment for this in the form of therapy and medication. Please talk to a professional.

I know intimacy is many things unrelated to sex but sometimes the fatigue overtakes me and I simply don’t care if I ever have sex. I also fall asleep when we try to be close.

Fatigue is a very common symptom of the MPNs, including the “lower-risk” MPNs including polycythemia vera and essential thrombocythemia. This can impede normal sexual activity and libido. One option is to try creative, non-medical, interventions. Think about what times of the day you are at your best. Perhaps timing your intimate encounters to be at a time of day when you feel more energized? Experiment with alterations in your diet – are there some foods that make you sleepy? If so, you can avoid those when you want to remain energized. Certainly, avoiding alcohol might help to prevent fatigue on “date nights.” A lack of libido can also be a sign of depression and certain antidepressants can help with this. I recommend discussing this all with your physician.